Amylyx will now work with federal, provincial, and territorial drug plans to ensure public coverage is available to eligible Canadians with ALS
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced that it has completed the negotiation process and entered into a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA) for the terms and conditions under which ALBRIOZA™ (sodium phenylbutyrate and ursodoxicoltaurine) would qualify for reimbursement through federal, provincial, and territorial public drug plans for the treatment of amyotrophic lateral sclerosis (ALS).
“Today marks a major milestone for people living with ALS and their caregivers in Canada. With this signed LOI, eligible Canadians living with this rapidly progressive disease who rely on public coverage are one step closer to accessing ALBRIOZA,” said Chris Aiello, General Manager and Head of Canada at Amylyx. “Our work is by no means done. We are now focused on the final task of translating this LOI into completed product listing agreements with each of the federal, provincial, and territorial jurisdictions to help ensure ALBRIOZA is listed on public formularies as soon as possible. This is a top priority given Canadians living with ALS simply do not have the time to wait.”
ALS is a relentlessly progressive and fatal neurodegenerative disease caused by motor neuron death in the brain and spinal cord that has a life expectancy of two to three years after symptom onset. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually death. The CENTAUR clinical trial demonstrated that ALBRIOZA significantly slowed disease progression and loss of functional decline in people living with ALS, potentially giving them more time with functional independence. ALBRIOZA received approval with conditions from Health Canada in June 2022, marking the first regulatory approval for ALBRIOZA issued to Amylyx worldwide.
“As a physician who treats people with ALS, access to treatments through public drug plans is critical for all those who may benefit,” said Dr. Angela Genge, Neurologist and Director of the ALS Clinic at the Montreal Neurological Institute-Hospital - The Neuro. “Being able to prescribe a new treatment that can help slow the progression of the disease and preserve physical function will greatly impact this population for whom there have been too few treatment options.”
“While it is good news that Canadians living with ALS are closer to accessing this innovative treatment, some people living with ALS have been waiting a year to access this therapy, and they will continue to wait,” said Tammy Moore, CEO of the ALS Society of Canada. “People with ALS do not have the luxury of time. We believe there are opportunities to streamline the multiple steps and lengthy processes involved in achieving public reimbursement for treatments like ALBRIOZA in Canada. The time is now for equitable and expedited access to approved treatments for ALS, and we will continue advocating on behalf of the ALS community to improve their access to care.”
With the LOI now concluded, Amylyx will turn to federal, provincial, and territorial public drug plans with a great sense of urgency to work on finalizing and signing product listing agreements for public funding of ALBRIOZA.
ALBRIOZA™ (sodium phenylbutyrate and ursodoxicoltaurine), previously known as AMX0035, is an oral fixed-dose medication approved with conditions to treat amyotrophic lateral sclerosis (ALS) in Canada. It is approved to treat ALS in adults in the U.S. as RELYVRIO®. Additionally, the Company’s Marketing Authorisation Application for AMX0035 for the treatment of ALS remains under review by the European Medicines Agency (EMA) in Europe. EMA recently informed the Company that the Committee for Medicinal Products for Human Use is trending toward a negative opinion on the AMX0035 application. AMX0035 is being explored for the potential treatment of other neurodegenerative diseases. The formulation of RELYVRIO, ALBRIOZA, and AMX0035 are identical.
About the CENTAUR Trial
CENTAUR was a multicenter Phase 2 clinical trial in 137 participants with ALS encompassing a 6-month randomized placebo-controlled phase and an open-label long-term follow-up phase. The trial met its primary efficacy endpoint of reducing functional decline as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale – Revised (ALSFRS-R).
Overall, reported rates of adverse events and discontinuations were similar between AMX0035 and placebo groups during the 24-week randomized phase; however, GI events occurred with greater frequency (≥2%) in the AMX0035 group. Detailed safety and functional efficacy data from CENTAUR were published in the New England Journal of Medicine. Data from additional analyses from the CENTAUR trial were published in Muscle & Nerve in 2020 and 2022, and the Journal of Neurology, Neurosurgery and Psychiatry in 2022.
The CENTAUR trial was funded, in part, by the ALS ACT grant and the ALS Ice Bucket Challenge, and was supported by The ALS Association, ALS Finding a Cure (a program of The Leandro P. Rizzuto Foundation), the Northeast ALS Consortium, and the Sean M. Healey & AMG Center for ALS at Mass General.
About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is committed to supporting and creating more moments for the neurodegenerative disease community through the discovery and development of innovative new treatments. Amylyx is headquartered in Cambridge, Massachusetts and has operations in Canada and EMEA. For more information, visit amylyx.ca and follow us on LinkedIn and Twitter.
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the Company’s plans to work with federal, provincial, and territorial drug plans in Canada in an effort to secure public coverage of ALBRIOZA; the potential of AMX0035 as a treatment for ALS and of the LOI to benefit Canadians living with ALS who rely on public coverage; beliefs around opportunities to streamline the processes involved in achieving public reimbursement in Canada; the potential continued market acceptance and market opportunity for ALBRIOZA; the EMA’s review of the Company’s Marketing Authorisation Application for AMX0035 for the treatment of ALS in Europe; the Company’s plans to explore the use of AMX0035 for other neurodegenerative diseases; and the Company’s priorities and expectations regarding its longer-term strategy. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: Amylyx’ ability to fund operations, the success, cost, and timing of Amylyx’ program development activities, Amylyx’ ability to execute on its commercial and regulatory strategy, regulatory developments, expectations regarding the timing and outcome of EMA’s review of AMX0035 for the treatment of ALS, Amylyx’ reliance on third parties, including to conduct clinical trials and manufacture products, and the effect of global economic uncertainty and financial market volatility caused by economic effects of rising inflation and interest rates, the COVID-19 pandemic, geopolitical instability, changes in international trade relationships and military conflicts, as well as the risks and uncertainties set forth in Amylyx’ United States Securities and Exchange Commission (SEC) filings, including Amylyx’ Quarterly Report on Form 10-Q for the quarter ended March 31, 2023, and subsequent filings with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Subject to any obligations under applicable law, Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.